The US Food and Drug Administration’s (FDA) Commissioner Scott Gottlieb said Monday the agency will advance a new framework to better regulate stem cell therapies this fall.

“This comprehensive policy will establish clearer lines around when these regenerative medicine products have sufficient complexity to fall under the agency’s current authority, and then define an efficient process for how these products should be evaluated for safety and effectiveness,” Gottlieb said.

The statement was sent out alongside a warning letter for a Florida stem cell clinic, known as US Stem Cell Clinic, LLC, and a news release on US Marshalls seizing five vials of a vaccine FDA says is reserved for people at high risk for smallpox, such as some military personnel.

The seizure of the vaccine vials from StemImmune, Inc. in California on Friday came as they were being used to create unapproved products that were injected intravenously into patients’ cancer tumors. StemImmune did not respond to a request for comment. US Stem Cell Clinic said in a press release that it is “not manufacturing a drug and therefore medical clinics are not required to follow cGMP.”

Gottlieb added: “The FDA will not allow deceitful actors to take advantage of vulnerable patients by purporting to have treatments or cures for serious diseases without any proof that they actually work. I especially won’t allow cases such as this one to go unchallenged, where we have good medical reasons to believe these purported treatments can actually harm patients and make their conditions worse.”

He added that the new policies will be set via a series of guidance documents to “give current product developers a very reasonable period of time” to interact with FDA to determine if they need to submit a marketing application.

He also said the agency is developing a novel approach to FDA approval of stem cell therapies that “we believe will allow very small product developers to gain all the benefits of FDA approval through a process that is minimally burdensome and less costly.”

But the comments and crackdown come as FDA has so far let these direct-to-consumer stem cell clinics flourish and has only issued a limited number of warning letters. Texas also recently passed a new law to allow companies to sell unproven stem cell treatments without FDA approval. Other clinics have been found in California and Florida, experts say.

Leigh Turner, an assistant professor of bioethics at the University of Minnesota, who has co-written an article in Cell about direct-to-consumer stem cell clinics, told Focus that FDA’s enforcement push and statements issued Monday seem to make the claim that there are a few bad actors but there are actually hundreds of these unscrupulous companies.

And though these companies “are not that difficult to find,” Turner said, “the bigger problem is once you find the businesses, what do you do, wait until there are patient complaints to issue a warning letter? Or do you issue a warning letter when dubious marketing claims are made?”

He noted that Florida’s US Stem Cell Clinic has been in operation for years and there is still a lack of clarity on “where the lines will be drawn” by FDA. And the StemImmune seizure is representative of “outrageous behavior,” he added, but “not a good example” of the larger issue.

credit: RAPS